PCSK9 Inhibitors Not Cost-effective at Current Price: ICER Review

September 09, 2015

WASHINGTON, DC — In order to be cost-effective for patients, providers, and payers, the new proprotein convertase subtilisin kexin 9 (PCSK9) inhibitors would need to cost around $2400 per year, a mark that is strikingly lower than what manufacturers are currently charging for the two available drugs, according to a new report[1].

Using a "willingness-to-pay" threshold of $50,000 per quality-adjusted life-year (QALY) gained, the PCSK9 inhibitors would need to cost $2100 per year for the familial hypercholesterolemia (FH) patient population. For secondary-prevention patients with LDL-cholesterol levels >70 mg/dL despite taking a maximally tolerated statin, a cost of $2400 per year would render the new monoclonal antibodies cost-effective. Finally, for statin-intolerant patients with CVD and an LDL-cholesterol level >70 mg/dL, the drugs would be cost-effective at the same willingness-to-pay threshold if priced at $2600 per year.

Even when different willingness-to-pay thresholds were used, including the generous $150,000 per QALY gained, the new drugs were cost-effective only if priced at a maximum of $5200 per year.

At present, evolocumab (Repatha, Amgen) and alirocumab (Praluent, Sanofi/Regeneron), both of which were approved by the US Food and Drug Administration this summer, cost $14,100 and $14,600, respectively, for a year-long supply of the LDL-cholesterol–lowering medication. In Europe, evolocumab, the only agent approved by the European Commission, costs $6800 per year in the UK and $8200 and $8800 per year in Austria and Finland, respectively.

The new report, conducted by the Institute for Clinical and Economic Review (ICER), an independent, nonprofit research group, evaluated the potential cost-effectiveness of the new drugs "to support dialogue needed for successful action to improve the quality and value of healthcare for all patients."

Massive Costs to US Healthcare System

The cost-effectiveness analysis is based on three target populations chosen to approximate the patients indicated for treatment with alirocumab, a drug that was approved earlier in the summer ahead of evolocumab (alirocumab and evolocumab have similar indications). The cost-effectiveness analysis was modeled on the cost of the drugs along with person-years of treatment, major adverse cardiovascular events (MACE) averted, and QALYs gained, among other variables.

Using the current manufacturer-set prices with a statin-treated FH patient as the comparator, the incremental cost-effectiveness ratio (ICER) of treatment with a statin plus a PCSK9 inhibitor was $681,000/QALY in the FH setting. In the secondary-prevention patient with a prior history of CVD (and statin intolerant), the ICER of adding a PCSK9 inhibitor vs no additional lipid-lowering therapy was $506,000/QALY. In a similar patient with an LDL-cholesterol level >70 mg/dL despite statin therapy, the ICER when adding a PCSK9 inhibitor vs a statin alone was $557,000/QALY.

For the reviewers, they estimate that approximately 2.6 million US individuals could potentially receive a PCSK9 inhibitor over the upcoming 5 years, which would have a total budgetary impact over that time period of $19 billion, $15 billion, and $74 billion for the FH, CVD/statin-intolerant, and CVD/not at LDL-cholesterol target subpopulations.

If the PCSK9 inhibitors were priced at $2412 per year and if only 50% of all eligible patients were treated over the 5-year period, this would add approximately $4 billion to the budget each year. Instead, if the cost of the drug were $14,600, but only 25% of eligible patients take a PCSK9 inhibitor, the cost to the healthcare budget would be $19 billion, "meaning that over the 5-year period a total of almost $100 billion would have been added to healthcare costs in the US."

Amgen Disagrees, Sanofi Still Analyzing the Analysis

Both alirocumab and evolocumab are very effective at lowering LDL-cholesterol levels, the reviewers concede, and data from a meta-analysis looking at clinical outcomes suggested a reduction in all-cause mortality and MI. They caution, however, the studies assessing hard outcomes were too short and underpowered. Moreover, other agents have been shown to significantly reduce LDL-cholesterol levels—torcetrapib, dalcetrapib, and niacin, among others—but have not reduced hard clinical end points. Outcome data from ODYSSEY and FOURIER will not be available until 2017 at the earliest.

In a statement to heartwire from Medscape, Amgen stated that it believes in the clinical and economic value of evolocumab, and while it welcomes the discussion, it "disagrees with ICER's methodology, assumptions, and preliminary conclusions, none of which have undergone public comment or formal peer review." The company says the review doesn't place value on addressing an unmet clinical need and will only serve to create unneeded barriers to patients who need the drug.

For its part, Sanofi also issued a statement to heartwire , saying that it needs to better understand the analysis, "particularly their model and methodology, as we performed our own cost-effectiveness model to help us determine the appropriate price." Sanofi added that statins will remain the cornerstone of treatment for lowering LDL cholesterol in the "vast majority of patients." Although alirocumab, as well as evolocumab, are available, they expect payers to ensure statins are "fully optimized, as many patients today are undertreated."

Sanofi added that it plans to provide more detailed feedback to ICER during the open comment period. From September 8 until September 22, ICER will hear comments from the public and revise the economic report in October. Full and complete findings will be presented at a meeting of the New England Comparative Effectiveness Public Advisory Council (CEPAC) on October 27, 2015 in Boston, MA.

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